Industry-Supported Symposia

Symposium Days:

Saturday, July 7, 2018

Room: Park 2
Luncheon Symposium supported by Sarepta
Precision-genetic therapies for DMD

  • Precision-genetic Therapy Approaches: An Overview – Perry Shieh
  • Exon-Skipping Therapies: Summary of Clinical Data – Eugenio Mercuri
  • RNA & Gene Therapy: Combination Approaches – Thomas Voit

To register, please click here.

Room: Park 1
Luncheon Symposium supported by CSL Behring
Subcutaneous Immunoglobulin in CIDP: Getting Under the Skin
Chair: Vera Bril

  • Expanding the Evidence for Subcutaneous Immunoglobulin in CIDP: PATH and Beyond – Hans Katzberg
  • Administering Subcutaneous Immunoglobulin: A Nurse’s Perspective – Dorothea Grosse-Kreul
  • Patient Experiences with Intravenous and Subcutaneous Immunoglobulin Therapy – James Babington Smith

Sunday, July 8, 2018

Room: Berg
Breakfast Symposium supported by LUPIN
A clinical approach in non-dystrophic and dystrophic myotonia
Chair: Giovanni Meola

  • The spectrum of myotonic dystrophies (DM) and non-dystrophic myotonias – Christiane Schneider-Gold
  • Outcome measure of myotonia – Giovanni Meola

Room: Park 2
Luncheon Symposium supported by Santhera Pharmaceuticals
Respiratory function decline in Duchenne muscular dystrophy (DMD) – Insights and evolving treatment strategies
Chair: Thomas Voit

  • New insights into the natural history of respiratory function decline in patients with DMD – Craig McDonald
  • Establishing clinically relevant thresholds of respiratory function decline and respiratory complications in DMD – Oscar H Mayer
  • Treatment strategies to slow respiratory function decline in DMD and address unmet needs – Thomas Voit

Room: Park 1
Luncheon Symposium supported by Biogen
Spinal Muscular Atrophy: Challenges Beyond Childhood
Chair: Janbernd Kirschner

  • SMA in teenagers and adults: our current understanding – Janbernd Kirschner
  • Considerations for optimizing care for teenage and adult patients with SMA – Maggie Walter
  • Living a normal life in an extraordinary way – Valeria Sansone

Monday, July 9, 2018

Room: Klimt 1
Breakfast Symposium supported by Alnylam Pharmaceuticals
Diagnosing and Assessing Burden of hATTR Amyloidosis: A Case Series
Chair: Isabel Conceição

  • A Case of Late Onset V30M: Delays in Diagnosis – Isabel Conceição
  • Challenges of hATTR Amyloidosis Diagnosis – Claudia Sommer
  • Recognizing Cardiac Manifestations of hATTR Amyloidosis – Pablo Garcia-Pavia

Room: Park 3 (room changed from Berg)
Breakfast Symposium supported by F. Hoffmann-La Roche Ltd.
SMA: balancing the emotional and clinical journey from molecule to medicine
Chair: Eugenio Mercuri

  • Challenges in the management of SMA: the patient and carer perspective (the emotional journey) – Nicole Gusset
  • From molecule to medicine (the clinical journey) – Laurent Servais  & Lutz Mueller

For more details, see here.

Room: Park 2
Luncheon Symposium supported by Alexion
A Novel, Targeted Treatment Option in Refractory Generalized Myasthenia Gravis
Chair: Renato Mantegazza

  • Disease Overview and the Patient Burden in Generalized Myasthenia Gravis – Renato Mantegazza
  • Role of Complement Inhibition in Patients with Anti-AChR+ gMG – Gil Wolfe
  • Patient Case Discussion Panel – Renato Mantegazza and Gil Wolfe

Click here to register and take an optional MG survey. The results will be discussed live during the symposium.

Room: Park 1
Luncheon Symposium supported by Sanofi Genzyme
Emerging Strategies in the diagnosis and management of muscle diseases
Chair: John Vissing

  • MYO-SEQ: experience from a large-scale next generation sequencing project of unclassified myopathies – Volker Straub
  • Unravelling patterns of muscle change in myopathies using MRI – Jordi Díaz-Manera
  • Treatment of muscle glycogenosis – John Vissing

Tuesday, July 10, 2018

Room: Park 1
Luncheon Symposium supported by PTC Therapeutics
Duchenne muscular dystrophy*: working together to reduce the time to diagnosis and improve patient outcomes
Chair: Günther Bernert

  • Striving for an earlier diagnosis of DMD: impact for patients and their families – Janbernd Kirschner
  • A look at the 2018 standards of care guidelines for DMD: is there a need for change in the current diagnostic and therapeutic procedures? – Günther Bernert
  • Ataluren* treatment for patients with nonsense mutation DMD: what do the latest data tell us? – Yann Péréon

* Ataluren is only indicated for the treatment of Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene, in ambulatory patients aged 5 years and older.

Room: Park 2
Luncheon Symposium provided by The France Foundation and supported by AveXis
Gene Replacement Therapy in Spinal Muscular Atrophy
Chair: Eugenio Mercuri

  • Richard S. Finkel
  • Gyula Acsadi


  • Genetics and Diagnostic Criteria of SMA
  • Emerging Data in SMA: Gene Replacement and Gene Modifying Therapies
  • Durability of New and Emerging Treatments
  • Implications for Standards of Care
  • Potential Applications to Other Neuromuscular Diseases

To register, click here.

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